Fiducial Lower Confidence Limit for The Reliability of Cold Standby System Consists of Geometric Series Components

2013 ◽  
Author(s):  
Lisheng Yang ◽  
Haiying Zheng
Keyword(s):  
Confidence Limit ◽  
Geometric Series ◽  
Lower Confidence Limit ◽  
Lower Confidence ◽  
Cold Standby ◽  
Standby System

scholarly journals Phase 2 study of subcutaneous omacetaxine mepesuccinate after TKI failure in patients with chronic-phase CML with T315I mutation

Blood ◽  
2012 ◽  
Vol 120 (13) ◽  
pp. 2573-2580 ◽  
Author(s):  
Jorge Cortes ◽  
Jeff H. Lipton ◽  
Delphine Rea ◽  
Raghunadharao Digumarti ◽  
Charles Chuah ◽  
...  
Keyword(s):  
Tyrosine Kinase ◽  
Confidence Limit ◽  
Chronic Phase ◽  
Cytogenetic Response ◽  
Phase 2 ◽  
Lower Confidence Limit ◽  
Hematologic Response ◽  
Lower Confidence ◽  
Omacetaxine Mepesuccinate ◽  
T315i Mutation

Abstract Chronic myeloid leukemia (CML) patients with the BCR-ABL T315I mutation do not benefit from therapy with currently approved tyrosine kinase inhibitors. Omacetaxine mepesuccinate is a protein synthesis inhibitor that has demonstrated activity in cells harboring the T315I mutation. This phase 2 trial assessed the efficacy of omacetaxine in CML patients with T315I and tyrosine kinase inhibitor failure. Patients received subcutaneous omacetaxine 1.25 mg/m2 twice daily, days 1-14, every 28 days until hematologic response or a maximum of 6 cycles, and then days 1-7 every 28 days as maintenance. Results for patients treated in chronic phase are reported here. Patients (n = 62) received a median of 7 (range, 1-41) cycles. Complete hematologic response was achieved in 48 patients (77%; 95% lower confidence limit, 65%); median response duration was 9.1 months. Fourteen patients (23%; 95% lower confidence limit, 13%) achieved major cytogenetic response, including complete cytogenetic response in 10 (16%). Median progression free-survival was 7.7 months. Grade 3/4 hematologic toxicity included thrombocytopenia (76%), neutropenia (44%), and anemia (39%) and was typically manageable by dose reduction. Nonhematologic adverse events were mostly grade 1/2 and included infection (42%), diarrhea (40%), and nausea (34%). Omacetaxine may provide a safe and effective treatment for CML patients with T315I mutation. This study is registered at www.clinicaltrials.gov as NCT00375219.

Sign in / Sign up

Export Citation Format

Share Document